JOP20210142A1 - Rnai constructs for inhibiting pnpla3 expression - Google Patents
Rnai constructs for inhibiting pnpla3 expressionInfo
- Publication number
- JOP20210142A1 JOP20210142A1 JOP/2021/0142A JOP20210142A JOP20210142A1 JO P20210142 A1 JOP20210142 A1 JO P20210142A1 JO P20210142 A JOP20210142 A JO P20210142A JO P20210142 A1 JOP20210142 A1 JO P20210142A1
- Authority
- JO
- Jordan
- Prior art keywords
- rnai constructs
- pnpla3 expression
- inhibiting pnpla3
- inhibiting
- liver disease
- Prior art date
Links
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- C—CHEMISTRY; METALLURGY
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1025—Acyltransferases (2.3)
- C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
- C12N9/20—Triglyceride splitting, e.g. by means of lipase
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y203/00—Acyltransferases (2.3)
- C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
- C12Y203/01051—1-Acylglycerol-3-phosphate O-acyltransferase (2.3.1.51)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01003—Triacylglycerol lipase (3.1.1.3)
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- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
The present invention relates to RNAi constructs for reducing expression of the PNPLA3 gene. Methods of using such RNAi constructs to treat or prevent liver disease, nonalcoholic fatty liver disease (NAFLD) are also described.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862777714P | 2018-12-10 | 2018-12-10 | |
| PCT/US2019/065481 WO2020123508A2 (en) | 2018-12-10 | 2019-12-10 | Rnai constructs for inhibiting pnpla3 expression and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| JOP20210142A1 true JOP20210142A1 (en) | 2023-01-30 |
Family
ID=69024749
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JOP/2021/0142A JOP20210142A1 (en) | 2018-12-10 | 2019-12-10 | Rnai constructs for inhibiting pnpla3 expression |
Country Status (23)
| Country | Link |
|---|---|
| US (1) | US20220017906A1 (en) |
| EP (1) | EP3894555A2 (en) |
| JP (2) | JP2022511550A (en) |
| KR (1) | KR20210102932A (en) |
| CN (1) | CN113166761B (en) |
| AR (1) | AR117297A1 (en) |
| AU (1) | AU2019396419A1 (en) |
| BR (1) | BR112021011061A2 (en) |
| CA (1) | CA3121510A1 (en) |
| CL (1) | CL2021001489A1 (en) |
| CO (1) | CO2021008997A2 (en) |
| CR (1) | CR20210371A (en) |
| EA (1) | EA202191629A1 (en) |
| IL (1) | IL283473A (en) |
| JO (1) | JOP20210142A1 (en) |
| MX (1) | MX2021006784A (en) |
| NZ (1) | NZ776661A (en) |
| PE (1) | PE20211225A1 (en) |
| PH (1) | PH12021551326A1 (en) |
| SG (1) | SG11202105711SA (en) |
| TW (1) | TW202039843A (en) |
| UY (1) | UY38503A (en) |
| WO (1) | WO2020123508A2 (en) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR113490A1 (en) * | 2017-12-12 | 2020-05-06 | Amgen Inc | RNAi CONSTRUCTIONS TO INHIBIT THE EXPRESSION OF PNPLA3 AND METHODS OF USE OF THE SAME |
| KR20220133248A (en) | 2020-01-28 | 2022-10-04 | 리제너론 파마슈티칼스 인코포레이티드 | Non-Human Animals Comprising Humanized PNPLA3 Locus and Methods of Use |
| WO2022221430A1 (en) | 2021-04-14 | 2022-10-20 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating pnpla3 expression |
| EP4347823A1 (en) * | 2021-06-02 | 2024-04-10 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| EP4402262A1 (en) * | 2021-09-01 | 2024-07-24 | Aligos Therapeutics, Inc. | Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof |
| JP2025524135A (en) * | 2022-07-27 | 2025-07-25 | イー-セラピューティクス・ピーエルシー | Nucleic acid compounds |
| EP4630556A1 (en) * | 2022-12-07 | 2025-10-15 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of patatin-like phospholipase domain-containing 3 (pnpla3) |
| AU2023408479A1 (en) * | 2022-12-19 | 2025-05-01 | Arnatar Therapeutics, Inc | Advanced rna targeting (arnatar) |
| WO2024173593A1 (en) * | 2023-02-14 | 2024-08-22 | Basecure Therapeutics | Modified double stranded rna agents |
| WO2024182446A2 (en) * | 2023-02-28 | 2024-09-06 | Aligos Therapeutics, Inc. | Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof |
| WO2024249328A2 (en) * | 2023-05-26 | 2024-12-05 | Adarx Pharmaceuticals, Inc. | Sod1-modulating compositions and methods of use thereof |
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| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
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| US7422902B1 (en) | 1995-06-07 | 2008-09-09 | The University Of British Columbia | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
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| AR113490A1 (en) * | 2017-12-12 | 2020-05-06 | Amgen Inc | RNAi CONSTRUCTIONS TO INHIBIT THE EXPRESSION OF PNPLA3 AND METHODS OF USE OF THE SAME |
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2019
- 2019-12-10 UY UY0001038503A patent/UY38503A/en not_active Application Discontinuation
- 2019-12-10 WO PCT/US2019/065481 patent/WO2020123508A2/en not_active Ceased
- 2019-12-10 US US17/312,721 patent/US20220017906A1/en active Pending
- 2019-12-10 MX MX2021006784A patent/MX2021006784A/en unknown
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| PE20211225A1 (en) | 2021-07-06 |
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| NZ776661A (en) | 2025-07-25 |
| US20220017906A1 (en) | 2022-01-20 |
| UY38503A (en) | 2020-06-30 |
| EP3894555A2 (en) | 2021-10-20 |
| CN113166761B (en) | 2025-09-09 |
| IL283473A (en) | 2021-07-29 |
| CL2021001489A1 (en) | 2022-01-07 |
| JP2024105284A (en) | 2024-08-06 |
| JP2022511550A (en) | 2022-01-31 |
| CA3121510A1 (en) | 2020-06-18 |
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